Gene Therapy for X-linked Severe Combined Immunodeficiency

Plain-language summary

This study examined a gene therapy (gene transfer) in children with X-linked severe combined immunodeficiency (SCID-X1). It enrolled boys diagnosed in the first year of life who had no suitable bone marrow donor and had severe infections. It was carried out in Phase 1/Phase 2 and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Completed

Sponsor

Assistance Publique - Hôpitaux de Paris

Intervention

Gene transfer

Start date

12/1/2010

Estimated completion

6/16/2015

Countries

France

Related genes

Locations

• Paris, France

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion criteria :

* Boys diagnosed during the first year of life
* Diagnosis of classical SCID-X1 based on immunophenotype (absent, or reduced numbers of non-functional T lymphocytes) and confirmed by DNA sequencing
* No HLA identical family donor and no HLA identical unrelated donor (10/10 antigens) found in the 6 weeks following the beginning of the search. This period could be shortened if the probability to find a donor is low or if the clinical situation (gravity) required
* Presence of a severe infection: pneumonitis and / or chronic diarrhea, or infection with herpes viruses or parainfluenza type 3 or adenovirus, or disseminated BCG infection, or presence of severe diarrhea and a severe compromise of the general state with denutrition
* Or failure of a HLA HAPLO-identical bone marrow transplant within 10 years after transplantation
* In all cases:

  * No family background of cancer in childhood.
  * No cytogenetic abnormalities (medullary karyotype) and no detection of main rearrangements associated with acute leukemia of children
  * Parental/guardian voluntary consent

Exclusion criteria :

* Atypical health with autologous T\> 500/ml3
* Infection by HIV 1 or 2
* Allogeneic HSC completed (excluding situations of failure)
* Existence of an HLA identical family donor or HLA identical unrelated donor
* No severe infections in a child with a preserved general state
* Family background of cancer in childhood
* Detection of cytogenetic abnormality and / or rearrangement associated with acute leukemia of children
* No affiliation to a social security scheme (beneficiary or assignee)